Crohn Disease
Conditions
Keywords
Crohn's Disease, GED-0301, Mongersen, Adolescent, Pediatric, Children
Brief summary
The purpose of study is to test the effect of an experimental medication GED-0301(mongersen) and evaluate its safety in patients (≥ 12 years of age) with active Crohn's disease. The study will test GED-0301 compare to placebo for 12 weeks. The study treatment is blinded which means that patients and the study doctor will not know which treatment has been assigned. Patients in this study will be allowed treatment with stable doses of oral aminosalicylates, oral corticosteroids, immunosupressants and antibiotics for the treatment of Crohn's disease. Adolescent patients will also be allowed treatment with stable doses of exclusive enteral nutrition and growth hormone. All patients who complete the study will have the option to enter a long term active treatment study.
Interventions
DRUGGED-0301
GED-0301
DRUGPlacebo
Placebo
Sponsors
Celgene
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Subjects must satisfy the following criteria to be enrolled in the study: * Male or female ≥ 12 years * Subject is able to swallow the IP tablets * Active Crohn's disease (CD) disease as determined by the Crohn's Disease Activity Index (CDAI) score and the Simple Endoscopic Score for Crohn's Disease (SES-CD) * Must meet a determined average minimum number of daily stools or rating of abdominal pain over a 7day period * Subject must have failed or experienced intolerance to at least one of the following: budesonide; systemic corticosteroids; immunosuppressants (eg, azathiopurine, 6-mercaptopurine, or methotrexate); or biologics for the treatment of CD (ie, infliximab, adalimumab, certolizumab, or vedolizumab).
Exclusion criteria
• The presence of any of the following will exclude a subject from enrollment: Diagnosis of ulcerative colitis (UC), indeterminate colitis, ischemic colitis, microscopic colitis, radiation colitis or diverticular disease-associated colitis, or colitis due to immunodeficiency * Local manifestations of Crohn's Disease (CD) such as abscesses, short bowel syndrome; or other disease complications for which surgery might be indicated or could confound the evaluation of efficacy * Subject has strictures with prestenotic dilatation, requiring procedural intervention, or with obstructive symptoms. In addition, subjects with colonic strictures that are not passable with an age-appropriate colonoscope, or strictures in the ileum or ileocecal valve that are fibrotic in nature, will be excluded * Intestinal resection within 6 months or any intra-abdominal surgery within 3 months prior to the Screening Visit * Ileostomy or a colostomy * Subject has a history of any clinically significant medical condition that, in the investigator's opinion, would prevent the subject from participating in the study * Adolescents with delayed growth or pubertal development who are on corticosteroids at baseline and who should not continue treatment with the same dose of corticosteroids until the Week 12 visit.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Clinical remission defined by an average daily liquid or soft stool frequency and abdominal pain score (US) and Crohn's Disease Activity Index (CDAI) (Rest of World) | week 12 | The proportion of subjects achieving clinical remission at Week 12. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Endoscopic improvement by the Simple Endoscopic Score for Crohn's Disease (SES-CD) | Week 12 | Endoscopic improvement of the mucosa at Week 12. |
| Clinical response defined by CDAI | Week 4, week 12 | The proportion of subjects achieving clinical response at Week 4, Week 12. |
| Clinical remission defined by an average daily liquid or soft stool frequency and abdominal pain score (US) and CDAI | Week 4, week 12 | The proportion of subjects achieving clinical remission at Week 4, Week 12. |
| Adverse Events (AEs) | Up to 20 weeks | Incidence and severity of adverse events |
| Pharmacokinetics (PK)- plasma concentration of GED-0301 | Week 4, week 8 | The plasma concentration of GED-0301 at Week 4, Week 8 |
| Clinical remission defined by PCDAI | Week 12 | The proportion of subjects achieving clinical remission at Week 12 (adolescent subjects only). |
Countries
Australia, Austria, Belgium, Canada, Croatia, Czechia, Denmark, Finland, France, Germany, Greece, Hungary, Israel, Italy, Latvia, Netherlands, Poland, Portugal, Romania, Russia, Serbia, Slovakia, South Korea, Spain, Sweden, Switzerland, Turkey (Türkiye), Ukraine, United Kingdom, United States
Outcome results
None listed